• Wave Life Sciences achieves first-ever RNA editing results in humans for alpha-1 antitrypsin deficiency, demonstrating therapeutic protein level increases with a single dose treatment.
• RNA editing emerges as a potentially safer alternative to DNA editing, with companies like Ascidian Therapeutics and Korro Bio advancing clinical trials for rare genetic disorders.
• The field has evolved significantly over the past decade, with new editing capabilities and delivery methods opening potential treatment opportunities for over 10 million patients.